Anne Zajicek, MD PharmD Chief (Acting) Obstetric and Pediatric Pharmacology Branch

1. Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Clinical Research Programs Anne Zajicek, MD PharmD Chief (Acting) Obstetric and Pediatric Pharmacology Branch Eunice Kennedy Shriver National Institute of Child Health and Human Development

2. Scope • The total annual NIH budget for pediatric research is about $ 3 billion • NICHD funds annually about 2000 clinical research projects with about 1700 Principal Investigators at about 500 institutions in 27 countries with a budget of about $ 650 million • The National Children’s Study has a separate budget that is administered through the NIH Director’s Office

3. NICHD Multi-Institution Support • NICHD either totally or partially supports about 60 clinical research consortia or networks annually • Operationally a consortium is multiple sites that receive support to conduct research on different aspects of a common topic or theme, share information and coordinate projects • Operationally a network is like a consortium with the additional dimension of sharing common multi-site protocols

4. Multi-Institution Projects • Currently about 60 % of the NICHD supported multi site projects are formal networks with shared protocols and about 40% are consortia working on a common theme • Most programs are on a 5 year cycle with the constituent institutions competitively reviewed every cycle, leading to a dynamic population • Entire programs are periodically evaluated with an ever evolving portfolio based on public health needs, scientific opportunities, available resources and other factors

5. Program Details and Listings • Additional information is listed on the Clinical Research section of the NICHD public web page http://www.nichd.nih.gov/health/clinicalresearch/NICHD.cfm

6. Examples of Extramurally Supported Clinical Research Networks Adolescent Literacy Research Network Adolescent Medicine Trials Network (ATN) for HIV/AIDS Interventions Adult Literacy Research Network Birth Defects Initiative and Research Network Child Health Research Centers Collaborative Pediatric Critical Care Research Network (CPCCRN) Community Child Health Research Network (CCHN) Contraceptive Clinical Trials Network (CCTN) Cooperative Research Program on Male Fertility Regulation Diabetes Research in Children Network (DirecNet) Domestic and International Pediatric/Perinatal HIV Clinical Studies Network Fragile X Syndrome Research Centers (FXRSC) Genomic and Proteomic Network on Premature Birth Research Global Network for Women’s and Children’s Health Research Intellectual and Developmental Disabilities Research Centers (IDDRCs)

7. Examples of Extramurally Supported Clinical Research Networks International Epidemiologic Database to Evaluate AIDS (IeDEA) International Maternal-Pediatric-Adolescent AIDS Clinical Trials (IMPAACT) Group Learning Disabilities Research Centers Maternal-Fetal Medicine Units Network Maternal-Fetal Surgery Network Mathematical Cognition and Math Disability Network Medical Rehabilitation Research Infrastructure Network Microbicide Trials Network (MTN) National Children's Study National Cooperative Program on Female Health and Egg Quality Neonatal Research Network NIAID/NICHD Primary Immunodeficiency Research Consortium NIH-DC Initiative to Reduce Infant Mortality in Minority Populations Obstetric-Fetal Pharmacology Research Unit (OPRU) Network

8. Examples of Extramurally Supported Clinical Research Networks Pediatric HIV/AIDS Cohort Study (PHACS) Pediatric Pharmacology Research Unit (PPRU) Network Pelvic Floor Disorders Network Prenatal Alcohol and SIDS and Stillbirth (PASS) Network Reproductive Medicine Network (RMN) Rare Disease Collaborative Research Centers Specialized Centers of Research (SCOR): Sex and Gender Issues affecting Women’s Health Specialized Cooperative Centers Program in Reproduction and Infertility Research (SCCPIR) Stillbirth Collaborative Research Network Study of Early Child Care and Youth Development (SECCYD) Traumatic Brain Injury (TBI) Clinical Trials Network Trial to Reduce the Incidence of Type 1 Diabetes for those Genetically at Risk (TRIGR) Urinary Incontinence Treatment Network (UITN) Women Interagency HIV Study (WIHS) Women and Infants Transmission Study (WITS) Work, Family, Health, and Well-Being Initiative

9. NICHD Partnerships • NICHD clinical research programs can be productive partnerships on many levels • Examples include partnerships with • NIH institutes for diabetes etiology and treatment, HIV infection, rare diseases, immunodeficiencies and other programs • National Cancer Institute for pediatric specific terminology and research informatics • Food and Drug Administration for identification of therapeutic needs and product labeling • Gates Foundation for tropical diseases and nutrition • Robert Wood Johnson Foundation for childhood obesity • Foundation for the National Institutes of Health and the Biomarker Consortium for identification of opportunities for biomarker development • District of Columbia for epidemiology of infant mortality • Government of India for clinical research and biotechnology development

10. Disparities on product use information for children • Historically pediatric populations have not been systematically included in the product development programs and subsequent product use information for FDA regulated products • Systematic inclusion of data in the product package insert (label) for drugs and biologics is relatively recent due to several Federal government initiatives • Most recent manifestation of the programs is in the Food and Drug Administration Amendments Act of 2007

11. NICHD Responsibilities • The NIH, in conjunction with the Food and Drug Administration, with NICHD as the lead, is required to perform a gap analysis and prioritize the need for information and the need for new therapeutic options for children based on disease or condition • The NICHD has additional responsibility to support programs to address information gaps for drug products when the private sector is unwilling or unable to provide the information • Applies to products that have patent or exclusivity protection and for products that are off patent and lack exclusivity • Coordinated effort with the Food and Drug Administration

12. Pediatric Device Development • NICHD is the NIH lead for coordinating and advancing medical device development for children • An interagency plan between the Agency for Healthcare Research and Quality, the Food and Drug Administration and the NIH mandated by Title III of the Food and Drug Administration Amendments Act of 2007is currently under departmental review • NIH has designated a central Point of Contact for device development

13. Current Funding Opportunity • In collaboration with the National Center for Research Resources, NICHD is funding an administrative supplement to the Clinical and Translational Science Awards for development of new pediatric outcome measures including age and developmentally specific clinical assessments, calibration of assessments across age groups, biomarker development and non-clinical predictive model development. • Priority areas are cardiology, neonatology and neurotoxicity; however, any proposal relevant to outcome measures in children can be submitted • Deadline is April 30, 2009

14. For further information • Food and Drug Administration Amendments Act of 2007 programs on pediatric drugs and prioritization Anne Zajicek, MD PharmD zajiceka@mail.nih.gov 301 435 6865 • For Administrative Supplement to CTSA sites on pediatric outcome measures • For process issues Mary Purucker, MD PhD puruckerm@mail.nih.gov 301 435 0741 • For scientific issues Dr. Anne Zajicek • For Pediatric Medical Devices and general information about NICHD clinical research Steven Hirschfeld, MD PhD hirschfs@mail.nih.gov 301 496 0044